99% Invisible Orphan Drugs
Nov 14, 2018
Dan Weissmann, a journalist and producer of An Arm and a Leg, dives into the fascinating world of orphan drugs—medications for rare diseases. He reveals the struggles faced by families, like Abby Myers with her son’s Tourette's, and how advocacy transformed into political momentum. A hit 70s TV show played a crucial role in raising awareness, leading to the landmark Orphan Drug Act of 1983. Weissmann also discusses the mixed legacy of the Act, highlighting soaring drug prices while also celebrating life-saving treatments for patients.
AI Snips
Chapters
Transcript
Episode notes
Mother Finds Treatment Through Chance
- Abby Myers discovered her son David had Tourette syndrome from a newspaper article and then found a clinical trial that helped him.
- The pharma company dropped the drug because schizophrenia was the target market, leaving the Tourette's treatment an orphan.
Learning To Lobby In Plain English
- Abby Myers joined the Tourette's association and learned advocacy from Marjorie Guthrie, who demanded plain English from experts.
- Guthrie used clear explanations to pin down pharma and regulators and turn technical answers into leverage.
TV Episode Sparks Real Advocacy
- A Los Angeles Times piece led a Quincy TV producer to contact Abby, which spawned a Quincy episode about orphan drugs.
- The broadcast generated thousands of letters and energized the movement for a real congressional hearing.